Summary and Direction Forward
The unintended consequences of well intentioned actions produced a situation in which children were therapeutic orphans, often denied the benefits of research and evidence in guiding therapy. While there has been considerable progress over the past two decades in addressing this issue, much remains to be done, notably as the next generation of therapeutics in the form of biologics, cell therapy and genetic engineering evolve. Fortunately there is will and energy, notably among the next generation of child health care providers and researchers, to address and surmount these challenges in pursuit of the common goal of effective and safe therapy for children everywhere.